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The pressing need for treatments to address neuromuscular disorders — which can affect the nerves that control both muscles and the communication between nerves and muscles — is helping to fuel related research.
Currently, only a limited number of drugs have been approved to treat the more than 30 challenging — and sometimes fatal — neuromuscular diseases; adequate options aren’t available for many of the conditions, and the drugs that do exist frequently produce multiple severe side effects, according to Technavio, such as sedation and paralysis.
Promising research, however, is underway, including gene and cell therapy that could potentially reverse neurological damage and enhance patients’ life expectancy; as a result, the analysis provider predicts the market for neuromuscular disease therapeutics will expand at a compound annual growth rate of 11.44% through 2028, ultimately increasing by more than $9 billion.
Technavio says the biologics segment had been the largest neuromuscular disease research area several years ago, once valued at $5.12 billion; rising gene therapy costs could threaten growth in the segment within the next four years — yet an increased prevalence of muscle and nerve problems, coupled with regulatory authorities approving new biologics, may help bolster biologics efforts.
In recent years, a number of institutional investors and hedge funds have expressed an interest in neuromuscular condition-related research.
Global value-oriented and event-driven hedge fund Armistice Capital, the British Marshall Wace LLP fund, and other entities, for example, have invested in Cytokinetics Incorporated, a late-stage biopharmaceutical organization that is investigating ways to treat impaired muscle function-related cardiovascular and neuromuscular diseases.
Cytokinetics says it has conducted some of the largest clinical trials relating to amyotrophic lateral sclerosis (ALS),formerly known as Lou Gehrig's disease — a disorder that, according to the National Institute of Neurological Disorders and Stroke, affects motor neurons and nerve cells in the brain and spinal cord that control voluntary muscle movement and breathing.
Cytokinetics’ research has involved reldesemtiv , an investigational, selective, small molecule fast skeletal muscle troponin activator (FSTA) that arose from the company’s skeletal muscle contractility program.
Cytokinetics isn’t the only ALS-related research effort hedge funds and institutional investors have supported in recent years.
In July of this year, MarketBeat reported that a number of investors had recently bought and sold shares of Zevra Therapeutics, Inc. — which discovers and develops proprietary prodrugs that address serious medical conditions.
During the fourth quarter of 2023, Armistice Capital purchased shares of Zevra. In the first quarter of the year, investment management company Vanguard Group Inc. added 58,866 shares of the company, increasing its position by 3.6%; and investment advisory firm Mystic Asset Management Inc. bought 35,514 more shares of Zevra, upping its stake to 15.4%, MarketBeat said.
Zevra Therapeutics has developed products such as AZSTARYS, which is used to treat attention Hyperactivity Disorder (ADHD) in patients age six and older. According to the company, its KP1077 drug — created to treat idiopathic hypersomnia and narcolepsy type I and II — is the leading candidate for addressing those conditions.
Zevra is also currently investigating treatments for neurological conditions such as Niemann-Pick disease type C (NPC), a very rare, progressive, and fatal genetic disorder that involves an inability to transport cholesterol and other fatty substances inside of cells, which leads to the substances accumulating within brain and other tissue in the body, damaging the affected areas, the National Organization for Rare Disorders says.
With a variable age of onset and symptoms, NPC is a challenging condition that can potentially be fatal in the first few months after birth — or present as a chronic disorder that isn’t diagnosed until adulthood.
In July of this year, Zevra announced the U.S. Food and Drug Administration (FDA) had told the company it is planning to meet with the recently formed Genetic Metabolic Diseases Advisory Committee (GeMDAC) in August to review the New Drug Application (NDA)for arimoclomol, Zevra’s orally delivered treatment for NPC.
Research involving neurological diseases — which, due to nervous system issues involving the brain, spinal cord, or nerves, can make moving, breathing and other actions difficult — has also drawn interest from institutional and other investors in 2024.
In March, for instance, CervoMed, a company that’s developing treatments for age-related neurologic disorders, entered into a private placement agreement led by investment manager RA Capital Management and other investors, such as healthcare investment firm Soleus Capital and Armistice Capital.
CervoMed agreed to sell an aggregate of 2,532,285 shares of its common stock and offer warrants to purchase common stock shares. The arrangement was expected to produce approximately $50 million in gross proceeds.
In a press release about the private placement agreement, CervoMed said it planned to use the funds on operating expenses and research relating to its clinical-stage neflamapimod medication, which treats central nervous system disorders such as strokes, dementia with Lewy bodies, and Alzheimer’s disease.
